Gene Editing Based Therapeutic Platforms
Ex Vivo Gene-editing Platform for Hematopoietic Stem Cells
The platform focuses on developing innovative therapies for β-thalassemia and other anemias based on the autologous hematopoietic stem cells through gene editing.
Ex Vivo Gene-editing Platforms for T Cells
The platform develops the allogeneic CAR-T therapies for cancer, that is to remove receptors and proteins that can cause adverse reactions such as GvH and HvG on T cells of a healthy donor.
In Vivo Therapeutic Platform Based on RNA Base Editing
The platform develops therapeutics for Usher syndrome type 2 (USH2), Hurler syndrome, the most severe subtype of mucopolysaccharidosis type I, diseases related to liver, muscles and nerve system, etc. based on the RNA base editing technology LEAPER™.